Abstract

  Background: Hemophilia-A is an X-linked genetic disorder arising from a mutation to the X chromosome and causing either a quantitative or qualitative deficiency in blood clotting factor-VIII (FVIII). Several recombinant extended half-life FVIII products (rFVIII-EHL) are in use in Italy for the treatment of hemophilia-A.  Objective: To estimate the annual average cost of the prophylactic treatment of a patient with hemophilia-A in Italy with rurioctocog alfa  pegol or turoctocog alfa pegol, with particular regard to potential dose adjustments as provided in the SPCs.  Methods: The comparison between the two rFVIII-EHL, expressed in terms of average annual cost per patient treated, was conducted from the NHS perspective. The average annual consumption (IU/kg) of the two rFVIII-EHL was derived from the clinical trial of reference for in label prophylactic treatment. Adjustments of doses and administration intervals allowed in the SPCs were considered on the basis of the literature. The published ex-factory price considered for both rFVIII-EHL was € 0.72. A scenario analysis focused on possible dosing adjustments was conducted.  Results: With regard to the average consumption associated with the posology in label, the average annual treatment cost is almost the same between the two rFVIII-EHL. Whereas taking into account the possible dose adjustments based on once-weekly administration, rurioctocog alfa pegol shows a considerably lower average annual treatment cost compared to turoctocog alfa pegol (– € 42,719).  Conclusions: Rurioctocog alfa pegol would be the more sustainable option, based on the annual average cost, for the NHS in the prophylactic treatment of hemophilia-A. 

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